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NCT Number: NCT03466463

Gene Therapy for Severe Crigler Najjar Syndrome

This is a Phase 1/2, multinational, open-label, study to evaluate the safety and efficacy of an intravenous infusion of GNT0003 in patients with Crigler-Najjar aged ≥10 years and requiring phototherapy. Patients will received a single administration of GNT0003 and will be followed for safety and efficacy of approximately 60 months (5 years):

* a follow-up of approximately 12 months (48 weeks) * a long term follow-up of approximately 48 months (4 years), in order to be in line with the latest EMEA Guideline on follow-up of patients administered with gene therapy medicinal products, released on 22 Oct.2009 by the Committee for medicinal products for human use.

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Key information

Conditions

Crigler-Najjar Syndrome Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hyperbilirubinemia, Hereditary Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases

Age range

9 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Hopital Antoine BECLERE, Clamart, France

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Patients with severe Crigler-Najjar syndrome resulting from a molecular confirmation of mutations in the UGT1A1 gene and requiring phototherapy
  • Male or female at least 9 years at the date of signature of informed consent
  • Patient able to give informed assent and/or consent in writing

Exclusion criteria

  • Patients who underwent liver transplantation
  • Patients with chronic hepatitis B or C
  • Patients infected with Human immunodeficiency virus (HIV)
  • Patients with significant underlying liver disease
  • Patients with significant encephalopathy
  • Participation in any other investigational trial during this trial
  • Patients unable or unwilling to comply with the protocol requirements

Treatment and study plan

GNT0003

Genetic

Intravenous infusion, single dose

Primary outcomes

  1. Proportion of patients having received the selected dose of GNT0003 with serum bilirubin ≤ 300µmol/L within 48 meeks after GNT0003 administration and without phototherapy from week 16

    Time frame: 48 weeks

    Decrease of total Serum bilirubin level after interruption of daily phototherapy (Efficacy); change in serum total biliirubin from baseline to week 48

  2. Incidence of Treatment Emergent Adverse Events or Treatement Serious Adverse Events

    Time frame: 48 weeks

    Incidence of AE/SAE evaluated by changes in laboratory parameters, vital signs, physical examination, reported from baseline to each visit study. Clinically relevant abnormal findings on Laboratory values, Vital Signs, Physical findings will be reported as Adverse Events.

    Incidence and Severity of Adverse Events for each body system will be presented for each dose level and summarized overall.

Secondary outcomes

  1. Change in Health-related quality of Life for Adults from Baseline to Week 48 after GNT0003 administration

    Time frame: 48 weeks

    Quality of Life outcome measure: change of quality of life from Baseline to Week 48 after GNT0003 administration.

    Scale 36-Item Short Form Survey (SF-36 Health Survey) for adults: from 0 (negative to health) to 100 (positive to health)

  2. Change in Health-related quality of Life for Children from Baseline to Week 48 after GNT0003 administration

    Time frame: 48 weeks

    Quality of Life outcome measure: change of quality of life from Baseline to Week 48 after GNT0003 administration.

    PedsQL 4.0 Generic Core Scale for pediatrics: 0-100 scale where higher scores indicate better health-related quality of life

Study contacts

Contact information is provided by the study sponsor or research team.

Genethon Clinical Development Department

CONTACT

[email protected]

00 33 (0)1 69 47 10 32

Sponsors and collaborators

Lead sponsor

Genethon

Other

Registry information

Official study title

A Phase I/II, Open Label, Study to Evaluate Safety and Efficacy of an Intravenous Injection of GNT0003 (AAV Vector Expressing the UGT1A1 Transgene) in Patients With Severe Crigler-Najjar Syndrome Requiring Phototherapy

Acronym: CareCN

Important dates

Study start
2018
Primary completion
2026
Study completion
2030
First posted
Mar 15, 2018
Registry last updated
Mar 28, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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